Researchers from the University of Sheffield in the UK are now predicting that a ddRNAi treatment for Motor Neurons Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS), will be ready for regulatory approval by August 2015. They say, "The experience that we have accumulated with our gene therapy and Clinical Trials programmes at SITraN is crucial to develop this new therapy for patients with ALS/MND. We plan submission for regulatory approval by August 2015, for permission to take this therapy to patients in the clinic. "
While this article does not say this work is ddRNAi, a little research reveals that Professor Mimoun Azzouz, who is mentioned in the article, published a paper back in 2006 on the subject. Furthermore, the ALS Advocate refers to Professor Pamela Shaw's work at University of Sheffield's Institute for Translational Neuroscience (SITraN) and her work is in silencing the SOD1 gene using AAV9 delivery of SOD1-shrna. These references confirm that this research is ddRNAi.
This team better get its skates on though because teams from the Research Institute at Nationwide Children's Hospital and the Ludwig Institute at the University of California, San Diego, are doing exactly the same work in their labs and they have already conducted studies in NHP.
It would seem that in a little over 12 months we could have another ddRNAi program or two moving into the clinical stage of development.
Both teams operate under the safe harbor provision of international patents and so no payments are due to Benitec (BNIKF, BTEBY) before commercialisation but the fact that the technology is progressing to clinical trial outside of Benitec's own pipeline is a big plus for the technology.
Disclosure: The author is long BNIKF.
Additional disclosure: This article is not intended as investment advice. Readers should due their own research.